Three of the lawmakers who will lead the House next year as Congress focuses on skyrocketing drug costs are among the biggest recipients of campaign contributions from the pharmaceutical industry, a new KHN analysis shows.

On Nov. 28, House Democrats selected Rep. Steny Hoyer of Maryland to serve as the next majority leader and Rep. James Clyburn of South Carolina as majority whip, making them the No. 2 and No. 3 most powerful Democrats as their party regains control of the House in January.

Both lawmakers have received more than $1 million from pharmaceutical company political action committees (PACs) in the past decade. Just four members of Congress hold that distinction, including Rep. Kevin McCarthy of California, whom Republicans chose as the next House minority leader earlier this month.

Adding Rep. Nancy Pelosi, the California Democrat expected to be the next speaker, the three-person House Democratic leadership team has collected more than $2.3 million total in campaign contributions from drugmakers since the 2007-2008 election cycle, according to KHN’s database.

High drug prices surfaced as a major campaign issue in 2018. With almost half of Americans saying they were worried about prescription drug costs last summer, many Democrats told voters they’d tackle the issue in the next Congress. But the large amount of money going to key Democrats, and Republicans, raises questions about whether Congress will take on the pharmaceutical industry.

In the past decade, members of Congress from both parties have received about $81 million from 68 pharma PACs run by employees of companies that make drugs and industry trade groups.

Brendan Fischer, who directs federal reform programs at the nonpartisan Campaign Legal Center, said drugmakers, like other wealthy industries, “shower money” on congressional leaders who are mulling legislation that could affect the pharmaceutical industry.

“Both Democrats and Republicans have discussed taking action on prescription drug prices, and drug companies likely expect that big contributions will help them maintain access to, and influence over, powerful lawmakers,” he said.

Mr. McCarthy, who has close ties to President Donald Trump, has received more than $1.08 million from drugmaker PACs since 2007. According to the latest data, which runs through September, he received about $250,000 this election cycle.

The fourth lawmaker to top $1 million is Sen. Richard Burr, a North Carolina Republican who serves on both the Senate Committee on Health, Education, Labor and Pensions and the Senate Committee on Finance. North Carolina is also home to a number of research universities and drugmakers’ headquarters.

While campaign contributions may seem tantalizing as a metric for influence, industries are not necessarily buying votes with their cash. More likely, they are buying access – a sizable donation from a drugmaker’s PAC may increase the chances its lobbyists get a meeting with an influential lawmaker, for example.

Mr. Clyburn, who like Mr. Hoyer has served as a top Democratic leader since 2007, has received more from drugmaker PACs over the past decade than any other member of Congress – more than $1.09 million. During the 2018 election cycle, he received at least $170,000, despite trouncing his Republican opponent in his safely Democratic district.

A party leader and the highest-ranking African-American in Congress, Mr. Clyburn has had ties to the pharmaceutical industry over the years. In 2013, he was a featured speaker at a conference hosted by PhRMA, the industry’s leading trade group. The conference was held at the James E. Clyburn Research Center at the Medical University of South Carolina, a hub for biopharmaceutical research.

This fall, Mr. Hoyer topped the million-dollar mark in drugmaker PAC contributions over the past decade, collecting more than $1.02 million since 2007 and more than $128,000 this election cycle.

“Mr. Hoyer’s positions on legislation are based on what is in the best interest of his constituents and the American people, and he has made it clear the new Congress will tackle rising health care and prescription drug costs,” said Mariel Saez, a Hoyer spokeswoman.

The offices of Mr. Clyburn, Mr. McCarthy, and Ms. Pelosi did not respond to requests for comment.

Ms. Pelosi, in contrast to her deputies, has received nearly $193,000 total from drugmaker PACs the past decade. In the month before the midterm elections, she intensified her calls for action to control drug prices, saying on Election Day that she believed Democrats could find “common ground” with Trump on addressing the problem.

Senior committee members also tend to draw huge sums from the industries they oversee. Rep. Frank Pallone of New Jersey, the Democrat who is expected to chair the House Committee on Energy and Commerce, received nearly $169,000 this election cycle from drugmaker PACs, according to KHN’s database. Since 2007, he has collected more than $840,000.

Similarly, Rep. Greg Walden, the Oregon Republican who is finishing his term as chair of the committee, received $302,300, the most of any member this election cycle in contributions from drugmaker PACs.

By contrast, Rep. Elijah Cummings – the Maryland Democrat who is expected to head the House Committee on Oversight and Government Reform – has attracted minimal drugmaker cash, receiving just $18,500 since the 2007-2008 election cycle. He has made it clear that he intends to target pharmaceutical companies next year as he investigates climbing drug costs.

Kaiser Health News is a nonprofit national health policy news service. It is an editorially independent program of the Henry J. Kaiser Family Foundation that is not affiliated with Kaiser Permanente.

Three of the lawmakers who will lead the House next year as Congress focuses on skyrocketing drug costs are among the biggest recipients of campaign contributions from the pharmaceutical industry, a new KHN analysis shows.

On Nov. 28, House Democrats selected Rep. Steny Hoyer of Maryland to serve as the next majority leader and Rep. James Clyburn of South Carolina as majority whip, making them the No. 2 and No. 3 most powerful Democrats as their party regains control of the House in January.

Both lawmakers have received more than $1 million from pharmaceutical company political action committees (PACs) in the past decade. Just four members of Congress hold that distinction, including Rep. Kevin McCarthy of California, whom Republicans chose as the next House minority leader earlier this month.

Adding Rep. Nancy Pelosi, the California Democrat expected to be the next speaker, the three-person House Democratic leadership team has collected more than $2.3 million total in campaign contributions from drugmakers since the 2007-2008 election cycle, according to KHN’s database.

High drug prices surfaced as a major campaign issue in 2018. With almost half of Americans saying they were worried about prescription drug costs last summer, many Democrats told voters they’d tackle the issue in the next Congress. But the large amount of money going to key Democrats, and Republicans, raises questions about whether Congress will take on the pharmaceutical industry.

In the past decade, members of Congress from both parties have received about $81 million from 68 pharma PACs run by employees of companies that make drugs and industry trade groups.

Brendan Fischer, who directs federal reform programs at the nonpartisan Campaign Legal Center, said drugmakers, like other wealthy industries, “shower money” on congressional leaders who are mulling legislation that could affect the pharmaceutical industry.

“Both Democrats and Republicans have discussed taking action on prescription drug prices, and drug companies likely expect that big contributions will help them maintain access to, and influence over, powerful lawmakers,” he said.

Mr. McCarthy, who has close ties to President Donald Trump, has received more than $1.08 million from drugmaker PACs since 2007. According to the latest data, which runs through September, he received about $250,000 this election cycle.

The fourth lawmaker to top $1 million is Sen. Richard Burr, a North Carolina Republican who serves on both the Senate Committee on Health, Education, Labor and Pensions and the Senate Committee on Finance. North Carolina is also home to a number of research universities and drugmakers’ headquarters.

While campaign contributions may seem tantalizing as a metric for influence, industries are not necessarily buying votes with their cash. More likely, they are buying access – a sizable donation from a drugmaker’s PAC may increase the chances its lobbyists get a meeting with an influential lawmaker, for example.

Mr. Clyburn, who like Mr. Hoyer has served as a top Democratic leader since 2007, has received more from drugmaker PACs over the past decade than any other member of Congress – more than $1.09 million. During the 2018 election cycle, he received at least $170,000, despite trouncing his Republican opponent in his safely Democratic district.

A party leader and the highest-ranking African-American in Congress, Mr. Clyburn has had ties to the pharmaceutical industry over the years. In 2013, he was a featured speaker at a conference hosted by PhRMA, the industry’s leading trade group. The conference was held at the James E. Clyburn Research Center at the Medical University of South Carolina, a hub for biopharmaceutical research.

This fall, Mr. Hoyer topped the million-dollar mark in drugmaker PAC contributions over the past decade, collecting more than $1.02 million since 2007 and more than $128,000 this election cycle.

“Mr. Hoyer’s positions on legislation are based on what is in the best interest of his constituents and the American people, and he has made it clear the new Congress will tackle rising health care and prescription drug costs,” said Mariel Saez, a Hoyer spokeswoman.

The offices of Mr. Clyburn, Mr. McCarthy, and Ms. Pelosi did not respond to requests for comment.

Ms. Pelosi, in contrast to her deputies, has received nearly $193,000 total from drugmaker PACs the past decade. In the month before the midterm elections, she intensified her calls for action to control drug prices, saying on Election Day that she believed Democrats could find “common ground” with Trump on addressing the problem.

Senior committee members also tend to draw huge sums from the industries they oversee. Rep. Frank Pallone of New Jersey, the Democrat who is expected to chair the House Committee on Energy and Commerce, received nearly $169,000 this election cycle from drugmaker PACs, according to KHN’s database. Since 2007, he has collected more than $840,000.

Similarly, Rep. Greg Walden, the Oregon Republican who is finishing his term as chair of the committee, received $302,300, the most of any member this election cycle in contributions from drugmaker PACs.

By contrast, Rep. Elijah Cummings – the Maryland Democrat who is expected to head the House Committee on Oversight and Government Reform – has attracted minimal drugmaker cash, receiving just $18,500 since the 2007-2008 election cycle. He has made it clear that he intends to target pharmaceutical companies next year as he investigates climbing drug costs.

Kaiser Health News is a nonprofit national health policy news service. It is an editorially independent program of the Henry J. Kaiser Family Foundation that is not affiliated with Kaiser Permanente.

Three of the lawmakers who will lead the House next year as Congress focuses on skyrocketing drug costs are among the biggest recipients of campaign contributions from the pharmaceutical industry, a new KHN analysis shows.

On Nov. 28, House Democrats selected Rep. Steny Hoyer of Maryland to serve as the next majority leader and Rep. James Clyburn of South Carolina as majority whip, making them the No. 2 and No. 3 most powerful Democrats as their party regains control of the House in January.

Both lawmakers have received more than $1 million from pharmaceutical company political action committees (PACs) in the past decade. Just four members of Congress hold that distinction, including Rep. Kevin McCarthy of California, whom Republicans chose as the next House minority leader earlier this month.

Adding Rep. Nancy Pelosi, the California Democrat expected to be the next speaker, the three-person House Democratic leadership team has collected more than $2.3 million total in campaign contributions from drugmakers since the 2007-2008 election cycle, according to KHN’s database.

High drug prices surfaced as a major campaign issue in 2018. With almost half of Americans saying they were worried about prescription drug costs last summer, many Democrats told voters they’d tackle the issue in the next Congress. But the large amount of money going to key Democrats, and Republicans, raises questions about whether Congress will take on the pharmaceutical industry.

In the past decade, members of Congress from both parties have received about $81 million from 68 pharma PACs run by employees of companies that make drugs and industry trade groups.

Brendan Fischer, who directs federal reform programs at the nonpartisan Campaign Legal Center, said drugmakers, like other wealthy industries, “shower money” on congressional leaders who are mulling legislation that could affect the pharmaceutical industry.

“Both Democrats and Republicans have discussed taking action on prescription drug prices, and drug companies likely expect that big contributions will help them maintain access to, and influence over, powerful lawmakers,” he said.

Mr. McCarthy, who has close ties to President Donald Trump, has received more than $1.08 million from drugmaker PACs since 2007. According to the latest data, which runs through September, he received about $250,000 this election cycle.

The fourth lawmaker to top $1 million is Sen. Richard Burr, a North Carolina Republican who serves on both the Senate Committee on Health, Education, Labor and Pensions and the Senate Committee on Finance. North Carolina is also home to a number of research universities and drugmakers’ headquarters.

While campaign contributions may seem tantalizing as a metric for influence, industries are not necessarily buying votes with their cash. More likely, they are buying access – a sizable donation from a drugmaker’s PAC may increase the chances its lobbyists get a meeting with an influential lawmaker, for example.

Mr. Clyburn, who like Mr. Hoyer has served as a top Democratic leader since 2007, has received more from drugmaker PACs over the past decade than any other member of Congress – more than $1.09 million. During the 2018 election cycle, he received at least $170,000, despite trouncing his Republican opponent in his safely Democratic district.

A party leader and the highest-ranking African-American in Congress, Mr. Clyburn has had ties to the pharmaceutical industry over the years. In 2013, he was a featured speaker at a conference hosted by PhRMA, the industry’s leading trade group. The conference was held at the James E. Clyburn Research Center at the Medical University of South Carolina, a hub for biopharmaceutical research.

This fall, Mr. Hoyer topped the million-dollar mark in drugmaker PAC contributions over the past decade, collecting more than $1.02 million since 2007 and more than $128,000 this election cycle.

“Mr. Hoyer’s positions on legislation are based on what is in the best interest of his constituents and the American people, and he has made it clear the new Congress will tackle rising health care and prescription drug costs,” said Mariel Saez, a Hoyer spokeswoman.

The offices of Mr. Clyburn, Mr. McCarthy, and Ms. Pelosi did not respond to requests for comment.

Ms. Pelosi, in contrast to her deputies, has received nearly $193,000 total from drugmaker PACs the past decade. In the month before the midterm elections, she intensified her calls for action to control drug prices, saying on Election Day that she believed Democrats could find “common ground” with Trump on addressing the problem.

Senior committee members also tend to draw huge sums from the industries they oversee. Rep. Frank Pallone of New Jersey, the Democrat who is expected to chair the House Committee on Energy and Commerce, received nearly $169,000 this election cycle from drugmaker PACs, according to KHN’s database. Since 2007, he has collected more than $840,000.

Similarly, Rep. Greg Walden, the Oregon Republican who is finishing his term as chair of the committee, received $302,300, the most of any member this election cycle in contributions from drugmaker PACs.

By contrast, Rep. Elijah Cummings – the Maryland Democrat who is expected to head the House Committee on Oversight and Government Reform – has attracted minimal drugmaker cash, receiving just $18,500 since the 2007-2008 election cycle. He has made it clear that he intends to target pharmaceutical companies next year as he investigates climbing drug costs.

Kaiser Health News is a nonprofit national health policy news service. It is an editorially independent program of the Henry J. Kaiser Family Foundation that is not affiliated with Kaiser Permanente.

CHICAGO – A single intra-articular injection of a novel drug known for now as SMO4690 resulted in statistically significant and clinically meaningful improvements in pain and physical function through 6 months of follow-up in a phase 2b study including 695 patients with moderately to severely symptomatic knee OA, Yusuf Yazici, MD, reported at the annual meeting of the American College of Rheumatology.

Bruce Jancin/MDedge News

Based on the encouraging results of this and another large phase 2 study, two pivotal phase 3, randomized clinical trials are due to start in the spring of 2019. If the results are positive, SMO4690 could become the first approved disease-modifying OA drug (DMOAD), something that’s been a long-sought, high-priority goal in rheumatology, noted Dr. Yazici, chief medical officer at San Diego–based Samumed, which is developing the drug, and a rheumatologist at New York University.

SMO4690 is a small molecule inhibitor of the Wnt signaling pathway. The drug has two distinct mechanisms of action for treatment of knee OA: It has an anti-inflammatory effect and it protects cartilage from degeneration, as demonstrated by a clinically significant improvement in joint space width by x-ray, compared with placebo at 12 months of follow-up after a single baseline injection in the earlier 455-patient, phase 2 study. Also, animal studies suggest SMO4690 generates cartilage, an exciting possibility now being evaluated in two ongoing serial MRI studies in knee OA patients.

Dr. Yazici presented patient-reported outcomes from the 695-patient, 24-week, multicenter, randomized, placebo-controlled, double-blind, phase 2b study, which evaluated four different concentrations of SMO4690. The 0.07- and 0.23-mg per 2-mL injection doses proved significantly better than placebo at 12 weeks – the primary endpoint – for all patient-reported outcomes.

The 0.23-mg dose of SMO4690 remained superior to placebo for all four patient-reported outcomes at subsequent assessments at weeks 16, 20, and 24. The 0.07-mg dose remained significantly better than placebo through week 20.

As in the earlier phase 2 study, SMO4690 raised no significant safety concerns. Adverse events were similar in type and frequency in the active-treatment and placebo groups.

The study was sponsored by Samumed, where Dr. Yazici is employed as chief medical officer.

bjancin@mdedge.com

SOURCE: Yazici Y et al. Arthritis Rheumatol. 2018;70(Suppl 10), Abstract L03.

CHICAGO – A single intra-articular injection of a novel drug known for now as SMO4690 resulted in statistically significant and clinically meaningful improvements in pain and physical function through 6 months of follow-up in a phase 2b study including 695 patients with moderately to severely symptomatic knee OA, Yusuf Yazici, MD, reported at the annual meeting of the American College of Rheumatology.

Bruce Jancin/MDedge News

Based on the encouraging results of this and another large phase 2 study, two pivotal phase 3, randomized clinical trials are due to start in the spring of 2019. If the results are positive, SMO4690 could become the first approved disease-modifying OA drug (DMOAD), something that’s been a long-sought, high-priority goal in rheumatology, noted Dr. Yazici, chief medical officer at San Diego–based Samumed, which is developing the drug, and a rheumatologist at New York University.

SMO4690 is a small molecule inhibitor of the Wnt signaling pathway. The drug has two distinct mechanisms of action for treatment of knee OA: It has an anti-inflammatory effect and it protects cartilage from degeneration, as demonstrated by a clinically significant improvement in joint space width by x-ray, compared with placebo at 12 months of follow-up after a single baseline injection in the earlier 455-patient, phase 2 study. Also, animal studies suggest SMO4690 generates cartilage, an exciting possibility now being evaluated in two ongoing serial MRI studies in knee OA patients.

Dr. Yazici presented patient-reported outcomes from the 695-patient, 24-week, multicenter, randomized, placebo-controlled, double-blind, phase 2b study, which evaluated four different concentrations of SMO4690. The 0.07- and 0.23-mg per 2-mL injection doses proved significantly better than placebo at 12 weeks – the primary endpoint – for all patient-reported outcomes.

The 0.23-mg dose of SMO4690 remained superior to placebo for all four patient-reported outcomes at subsequent assessments at weeks 16, 20, and 24. The 0.07-mg dose remained significantly better than placebo through week 20.

As in the earlier phase 2 study, SMO4690 raised no significant safety concerns. Adverse events were similar in type and frequency in the active-treatment and placebo groups.

The study was sponsored by Samumed, where Dr. Yazici is employed as chief medical officer.

bjancin@mdedge.com

SOURCE: Yazici Y et al. Arthritis Rheumatol. 2018;70(Suppl 10), Abstract L03.

CHICAGO – A single intra-articular injection of a novel drug known for now as SMO4690 resulted in statistically significant and clinically meaningful improvements in pain and physical function through 6 months of follow-up in a phase 2b study including 695 patients with moderately to severely symptomatic knee OA, Yusuf Yazici, MD, reported at the annual meeting of the American College of Rheumatology.

Bruce Jancin/MDedge News

Based on the encouraging results of this and another large phase 2 study, two pivotal phase 3, randomized clinical trials are due to start in the spring of 2019. If the results are positive, SMO4690 could become the first approved disease-modifying OA drug (DMOAD), something that’s been a long-sought, high-priority goal in rheumatology, noted Dr. Yazici, chief medical officer at San Diego–based Samumed, which is developing the drug, and a rheumatologist at New York University.

SMO4690 is a small molecule inhibitor of the Wnt signaling pathway. The drug has two distinct mechanisms of action for treatment of knee OA: It has an anti-inflammatory effect and it protects cartilage from degeneration, as demonstrated by a clinically significant improvement in joint space width by x-ray, compared with placebo at 12 months of follow-up after a single baseline injection in the earlier 455-patient, phase 2 study. Also, animal studies suggest SMO4690 generates cartilage, an exciting possibility now being evaluated in two ongoing serial MRI studies in knee OA patients.

Dr. Yazici presented patient-reported outcomes from the 695-patient, 24-week, multicenter, randomized, placebo-controlled, double-blind, phase 2b study, which evaluated four different concentrations of SMO4690. The 0.07- and 0.23-mg per 2-mL injection doses proved significantly better than placebo at 12 weeks – the primary endpoint – for all patient-reported outcomes.

The 0.23-mg dose of SMO4690 remained superior to placebo for all four patient-reported outcomes at subsequent assessments at weeks 16, 20, and 24. The 0.07-mg dose remained significantly better than placebo through week 20.

As in the earlier phase 2 study, SMO4690 raised no significant safety concerns. Adverse events were similar in type and frequency in the active-treatment and placebo groups.

The study was sponsored by Samumed, where Dr. Yazici is employed as chief medical officer.

bjancin@mdedge.com

SOURCE: Yazici Y et al. Arthritis Rheumatol. 2018;70(Suppl 10), Abstract L03.

Given its limited systemic toxicity, topical anthralin is an acceptable second-line treatment option for pediatric alopecia areata (AA), according to Sean Z. Wu, MD, of the department of dermatology, University of Cincinnati, and his associates.

In a retrospective study of 37 pediatric patients with AA, published in Pediatric Dermatology, Dr. Wu and his colleagues found that almost two-thirds experienced at least 50% regrowth of hair with topical anthralin treatment, but they described severe dermatitis and relapses as “potential drawbacks” of treatment.

The 37 patients were in the Cleveland Clinic AA areata database and began treatment with anthralin between 2004 and 2015, at aged 2-17 years (mean age 9). Over half (22) were females and most (31) were white. About 65% had patchy AA; the remainder had either alopecia totalis or alopecia universalis. Prior treatments included topical corticosteroids, minoxidil, and intralesional corticosteroids; four patients had not been treated previously. Patients were followed up from 51 days to more than 10 years, with a mean duration of 2.5 years. Treatment regimens, titrated up to achieve a mild to moderate dermatitis, included application of 0.5% cream for 5 minutes twice a week up to 1.0% cream for 30 minutes a day.

With topical anthralin, 12 (32%) of patients had complete scalp regrowth, 25 (68%) experienced at least 50% regrowth, and 5 (14%) had no response; in five patients, no follow-up information was available. Among those with at least 50% regrowth, the initial response was first noted at a mean of 3.4 months, and the mean time to maximal response was 15 months. This timeline suggests that treatment with anthralin should be continued beyond 1 year to ensure maximum beneficial results with hair regrowth, the authors wrote.

Factors associated with a positive response to treatment included less than 50% of scalp involvement. The two patients who used anthralin as monotherapy did not achieve a 50% scalp response, but the four treatment-naive patients were among those with at least 50% scalp regrowth, versus three of the five patients (60%) who had been treated previously with systemic steroids.

Two potential clinical limitations were noted during the study. Four patients had to stop treatment because of dermatitis, which suggests that patients and parents should be counseled about the potential for severe skin irritation with this treatment, the authors said. And among those who achieved at least 50% scalp regrowth, 16 of the 25 (64%) relapsed. The authors speculated that the effects of the drug could be temporary “or that the disease process may be able to overcome the anthralin effect over time.”

Dr. Wu and his coauthors cited the retrospective design and the small population size as major limitations of the study. Because some patients continued other treatments, it is “difficult to attribute scalp regrowth entirely to anthralin,” and variations in formulation and in treatment regimens are also factors to be considered, they cautioned. That AA has been found to spontaneously resolve, depending upon the severity of the disease, presents additional challenges for clinicians attempting to determine the extent to which anthralin offers therapeutic benefit, they pointed out.

In spite of the drug’s limitations, the authors concluded that the treatment was a safe and useful option as an “adjunct for those who fail first-line therapy with topical or intralesional corticosteroids.” They added that more work is needed to tailor treatment formulation, frequency, and duration to the specific needs of pediatric patients.

The authors had no relevant financial disclosures to report.

SOURCE: Wu SZ et al. Pediatr Dermatol. 2018;35:817-20.

Given its limited systemic toxicity, topical anthralin is an acceptable second-line treatment option for pediatric alopecia areata (AA), according to Sean Z. Wu, MD, of the department of dermatology, University of Cincinnati, and his associates.

In a retrospective study of 37 pediatric patients with AA, published in Pediatric Dermatology, Dr. Wu and his colleagues found that almost two-thirds experienced at least 50% regrowth of hair with topical anthralin treatment, but they described severe dermatitis and relapses as “potential drawbacks” of treatment.

The 37 patients were in the Cleveland Clinic AA areata database and began treatment with anthralin between 2004 and 2015, at aged 2-17 years (mean age 9). Over half (22) were females and most (31) were white. About 65% had patchy AA; the remainder had either alopecia totalis or alopecia universalis. Prior treatments included topical corticosteroids, minoxidil, and intralesional corticosteroids; four patients had not been treated previously. Patients were followed up from 51 days to more than 10 years, with a mean duration of 2.5 years. Treatment regimens, titrated up to achieve a mild to moderate dermatitis, included application of 0.5% cream for 5 minutes twice a week up to 1.0% cream for 30 minutes a day.

With topical anthralin, 12 (32%) of patients had complete scalp regrowth, 25 (68%) experienced at least 50% regrowth, and 5 (14%) had no response; in five patients, no follow-up information was available. Among those with at least 50% regrowth, the initial response was first noted at a mean of 3.4 months, and the mean time to maximal response was 15 months. This timeline suggests that treatment with anthralin should be continued beyond 1 year to ensure maximum beneficial results with hair regrowth, the authors wrote.

Factors associated with a positive response to treatment included less than 50% of scalp involvement. The two patients who used anthralin as monotherapy did not achieve a 50% scalp response, but the four treatment-naive patients were among those with at least 50% scalp regrowth, versus three of the five patients (60%) who had been treated previously with systemic steroids.

Two potential clinical limitations were noted during the study. Four patients had to stop treatment because of dermatitis, which suggests that patients and parents should be counseled about the potential for severe skin irritation with this treatment, the authors said. And among those who achieved at least 50% scalp regrowth, 16 of the 25 (64%) relapsed. The authors speculated that the effects of the drug could be temporary “or that the disease process may be able to overcome the anthralin effect over time.”

Dr. Wu and his coauthors cited the retrospective design and the small population size as major limitations of the study. Because some patients continued other treatments, it is “difficult to attribute scalp regrowth entirely to anthralin,” and variations in formulation and in treatment regimens are also factors to be considered, they cautioned. That AA has been found to spontaneously resolve, depending upon the severity of the disease, presents additional challenges for clinicians attempting to determine the extent to which anthralin offers therapeutic benefit, they pointed out.

In spite of the drug’s limitations, the authors concluded that the treatment was a safe and useful option as an “adjunct for those who fail first-line therapy with topical or intralesional corticosteroids.” They added that more work is needed to tailor treatment formulation, frequency, and duration to the specific needs of pediatric patients.

The authors had no relevant financial disclosures to report.

SOURCE: Wu SZ et al. Pediatr Dermatol. 2018;35:817-20.

Given its limited systemic toxicity, topical anthralin is an acceptable second-line treatment option for pediatric alopecia areata (AA), according to Sean Z. Wu, MD, of the department of dermatology, University of Cincinnati, and his associates.

In a retrospective study of 37 pediatric patients with AA, published in Pediatric Dermatology, Dr. Wu and his colleagues found that almost two-thirds experienced at least 50% regrowth of hair with topical anthralin treatment, but they described severe dermatitis and relapses as “potential drawbacks” of treatment.

The 37 patients were in the Cleveland Clinic AA areata database and began treatment with anthralin between 2004 and 2015, at aged 2-17 years (mean age 9). Over half (22) were females and most (31) were white. About 65% had patchy AA; the remainder had either alopecia totalis or alopecia universalis. Prior treatments included topical corticosteroids, minoxidil, and intralesional corticosteroids; four patients had not been treated previously. Patients were followed up from 51 days to more than 10 years, with a mean duration of 2.5 years. Treatment regimens, titrated up to achieve a mild to moderate dermatitis, included application of 0.5% cream for 5 minutes twice a week up to 1.0% cream for 30 minutes a day.

With topical anthralin, 12 (32%) of patients had complete scalp regrowth, 25 (68%) experienced at least 50% regrowth, and 5 (14%) had no response; in five patients, no follow-up information was available. Among those with at least 50% regrowth, the initial response was first noted at a mean of 3.4 months, and the mean time to maximal response was 15 months. This timeline suggests that treatment with anthralin should be continued beyond 1 year to ensure maximum beneficial results with hair regrowth, the authors wrote.

Factors associated with a positive response to treatment included less than 50% of scalp involvement. The two patients who used anthralin as monotherapy did not achieve a 50% scalp response, but the four treatment-naive patients were among those with at least 50% scalp regrowth, versus three of the five patients (60%) who had been treated previously with systemic steroids.

Two potential clinical limitations were noted during the study. Four patients had to stop treatment because of dermatitis, which suggests that patients and parents should be counseled about the potential for severe skin irritation with this treatment, the authors said. And among those who achieved at least 50% scalp regrowth, 16 of the 25 (64%) relapsed. The authors speculated that the effects of the drug could be temporary “or that the disease process may be able to overcome the anthralin effect over time.”

Dr. Wu and his coauthors cited the retrospective design and the small population size as major limitations of the study. Because some patients continued other treatments, it is “difficult to attribute scalp regrowth entirely to anthralin,” and variations in formulation and in treatment regimens are also factors to be considered, they cautioned. That AA has been found to spontaneously resolve, depending upon the severity of the disease, presents additional challenges for clinicians attempting to determine the extent to which anthralin offers therapeutic benefit, they pointed out.

In spite of the drug’s limitations, the authors concluded that the treatment was a safe and useful option as an “adjunct for those who fail first-line therapy with topical or intralesional corticosteroids.” They added that more work is needed to tailor treatment formulation, frequency, and duration to the specific needs of pediatric patients.

The authors had no relevant financial disclosures to report.

SOURCE: Wu SZ et al. Pediatr Dermatol. 2018;35:817-20.

Infant mortality in the United Sates dropped very slightly from 2015 to 2016 and has not changed significantly since 2011, according to the National Center for Health Statistics.

Overall infant mortality was 5.87 per 1,000 live births in 2016, which was not significantly less than the 2015 rate of 5.90 per 1,000 or the rate of 6.07 per 1,000 recorded in 2011, the NCHS said in a recent Data Brief. The rate for 2016 works out to 3.88 per 1,000 for the neonatal period (0-27 days) and 1.99 per 1,000 during the postneonatal period (28-364 days).

Maternal age had a significant effect on infant mortality in 2016. The rate was lowest for mothers aged 30-34 years (4.86 per 1,000) and highest for those under 20 years (8.69). All overall rates by maternal age were significantly different from each other, except for those of mothers aged 20-24 years (7.13) and those aged 40 years and over (7.27). Neonatal mortality was highest for the under-20 group and the 40-and-over group at 5.32 per 1,000, with the difference between them coming during the postneonatal period: 3.36 for those under 20 and 1.95 for the 40-and-overs, the NCHS investigators reported based on data from the National Vital Statistics System.

The leading cause of death during the neonatal period in 2016 was low birth weight at 98 per 1,000 live births, with congenital malformations second at 86 per 1,000. The leading cause of death in the postneonatal period was congenital malformations at 36 per 1,000, followed by sudden infant death syndrome (35 per 1,000), unintentional injuries (27 per 1,000), diseases of the circulatory system (9 per 1,000), and homicide (6 per 1,000), they added.

rfranki@mdedge.com

Infant mortality in the United Sates dropped very slightly from 2015 to 2016 and has not changed significantly since 2011, according to the National Center for Health Statistics.

Overall infant mortality was 5.87 per 1,000 live births in 2016, which was not significantly less than the 2015 rate of 5.90 per 1,000 or the rate of 6.07 per 1,000 recorded in 2011, the NCHS said in a recent Data Brief. The rate for 2016 works out to 3.88 per 1,000 for the neonatal period (0-27 days) and 1.99 per 1,000 during the postneonatal period (28-364 days).

Maternal age had a significant effect on infant mortality in 2016. The rate was lowest for mothers aged 30-34 years (4.86 per 1,000) and highest for those under 20 years (8.69). All overall rates by maternal age were significantly different from each other, except for those of mothers aged 20-24 years (7.13) and those aged 40 years and over (7.27). Neonatal mortality was highest for the under-20 group and the 40-and-over group at 5.32 per 1,000, with the difference between them coming during the postneonatal period: 3.36 for those under 20 and 1.95 for the 40-and-overs, the NCHS investigators reported based on data from the National Vital Statistics System.

The leading cause of death during the neonatal period in 2016 was low birth weight at 98 per 1,000 live births, with congenital malformations second at 86 per 1,000. The leading cause of death in the postneonatal period was congenital malformations at 36 per 1,000, followed by sudden infant death syndrome (35 per 1,000), unintentional injuries (27 per 1,000), diseases of the circulatory system (9 per 1,000), and homicide (6 per 1,000), they added.

rfranki@mdedge.com

Infant mortality in the United Sates dropped very slightly from 2015 to 2016 and has not changed significantly since 2011, according to the National Center for Health Statistics.

Overall infant mortality was 5.87 per 1,000 live births in 2016, which was not significantly less than the 2015 rate of 5.90 per 1,000 or the rate of 6.07 per 1,000 recorded in 2011, the NCHS said in a recent Data Brief. The rate for 2016 works out to 3.88 per 1,000 for the neonatal period (0-27 days) and 1.99 per 1,000 during the postneonatal period (28-364 days).

Maternal age had a significant effect on infant mortality in 2016. The rate was lowest for mothers aged 30-34 years (4.86 per 1,000) and highest for those under 20 years (8.69). All overall rates by maternal age were significantly different from each other, except for those of mothers aged 20-24 years (7.13) and those aged 40 years and over (7.27). Neonatal mortality was highest for the under-20 group and the 40-and-over group at 5.32 per 1,000, with the difference between them coming during the postneonatal period: 3.36 for those under 20 and 1.95 for the 40-and-overs, the NCHS investigators reported based on data from the National Vital Statistics System.

The leading cause of death during the neonatal period in 2016 was low birth weight at 98 per 1,000 live births, with congenital malformations second at 86 per 1,000. The leading cause of death in the postneonatal period was congenital malformations at 36 per 1,000, followed by sudden infant death syndrome (35 per 1,000), unintentional injuries (27 per 1,000), diseases of the circulatory system (9 per 1,000), and homicide (6 per 1,000), they added.

rfranki@mdedge.com

Using data from the Million Veteran Program (MVP), researchers found people with mutations for PDE3B, PCSK9, and ANGPTL4 had better cholesterol and triglyceride levels than did those without the mutations.

The PDE3B mutation seems to protect against heart disease. A PCSK9 mutation may reduce the risk not only of heart disease, but also abdominal aortic aneurysm. The ANGPTL4 mutation was linked to lower risk of type 2 DM.

MVP, 1 of the world’s largest databases of health and genomic information, partners with veterans receiving care in the VHA to study how genes affect health. It has enrolled > 700,000 veterans to date.

Using data from the Million Veteran Program (MVP), researchers found people with mutations for PDE3B, PCSK9, and ANGPTL4 had better cholesterol and triglyceride levels than did those without the mutations.

The PDE3B mutation seems to protect against heart disease. A PCSK9 mutation may reduce the risk not only of heart disease, but also abdominal aortic aneurysm. The ANGPTL4 mutation was linked to lower risk of type 2 DM.

MVP, 1 of the world’s largest databases of health and genomic information, partners with veterans receiving care in the VHA to study how genes affect health. It has enrolled > 700,000 veterans to date.

Using data from the Million Veteran Program (MVP), researchers found people with mutations for PDE3B, PCSK9, and ANGPTL4 had better cholesterol and triglyceride levels than did those without the mutations.

The PDE3B mutation seems to protect against heart disease. A PCSK9 mutation may reduce the risk not only of heart disease, but also abdominal aortic aneurysm. The ANGPTL4 mutation was linked to lower risk of type 2 DM.

MVP, 1 of the world’s largest databases of health and genomic information, partners with veterans receiving care in the VHA to study how genes affect health. It has enrolled > 700,000 veterans to date.

Physical activity may count more for women who keep the pounds off. Also today, there may be a link between vitamin D levels and atopic dermatitis in children, nerve growth factor antibody cuts osteoarthritis pain with low adverse effects, and how you can harness the power of the urine test in pain care.

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Physical activity may count more for women who keep the pounds off. Also today, there may be a link between vitamin D levels and atopic dermatitis in children, nerve growth factor antibody cuts osteoarthritis pain with low adverse effects, and how you can harness the power of the urine test in pain care.

Amazon Alexa
Apple Podcasts
Google Podcasts
Spotify

Physical activity may count more for women who keep the pounds off. Also today, there may be a link between vitamin D levels and atopic dermatitis in children, nerve growth factor antibody cuts osteoarthritis pain with low adverse effects, and how you can harness the power of the urine test in pain care.

Amazon Alexa
Apple Podcasts
Google Podcasts
Spotify

Astellas Pharma

The U.S. Food and Drug Administration (FDA) has approved gilteritinib (Xospata) for use in adults who have relapsed or refractory acute myeloid leukemia (AML) with a FLT3 mutation, as detected by an FDA-approved test.

The FDA also expanded the approved indication for the LeukoStrat CDx FLT3 Mutation Assay to include use with gilteritinib.

The LeukoStrat CDx FLT3 Mutation Assay, developed by Invivoscribe Technologies, Inc., is used to detect FLT3 mutations in patients with AML.

Gilteritinib, developed by Astellas Pharma, has demonstrated inhibitory activity against FLT3 internal tandem duplication (ITD) and FLT3 tyrosine kinase domain.

The FDA’s approval of gilteritinib was based on an interim analysis of the ADMIRAL trial (NCT02421939).

The trial enrolled adults with relapsed or refractory AML who had a FLT3 ITD, D835, or I836 mutation, according to the LeukoStrat CDx FLT3 Mutation Assay.

Patients received gilteritinib at 120 mg daily until they developed unacceptable toxicity or did not show a clinical benefit.

Efficacy results are available for 138 patients, with a median follow-up of 4.6 months (range, 2.8 to 15.8).

The complete response (CR) rate was 11.6% (16/138), the rate of CR with partial hematologic recovery (CRh) was 9.4% (13/138), and the rate of CR/CRh was 21% (29/138).

The median duration of CR/CRh was 4.6 months.

There were 106 patients who were transfusion-dependent at baseline, and 33 of these patients (31.1%) became transfusion-independent during the post-baseline period.

Seventeen of the 32 patients (53.1%) who were transfusion-independent at baseline remained transfusion-independent.

Safety results are available for 292 patients. The median duration of exposure to gilteritinib in this group was 3 months (range, 0.1 to 42.8).

The most common adverse events were myalgia/arthralgia (42%), transaminase increase (41%), fatigue/malaise (40%), fever (35%), noninfectious diarrhea (34%), dyspnea (34%), edema (34%), rash (30%), pneumonia (30%), nausea (27%), constipation (27%), stomatitis (26%), cough (25%), headache (21%), hypotension (21%), dizziness (20%), and vomiting (20%).

Eight percent of patients (n=22) discontinued gilteritinib due to adverse events. The most common were pneumonia (2%), sepsis (2%), and dyspnea (1%).

For more details on the ADMIRAL trial and gilteritinib, see the full prescribing information.

Astellas Pharma

The U.S. Food and Drug Administration (FDA) has approved gilteritinib (Xospata) for use in adults who have relapsed or refractory acute myeloid leukemia (AML) with a FLT3 mutation, as detected by an FDA-approved test.

The FDA also expanded the approved indication for the LeukoStrat CDx FLT3 Mutation Assay to include use with gilteritinib.

The LeukoStrat CDx FLT3 Mutation Assay, developed by Invivoscribe Technologies, Inc., is used to detect FLT3 mutations in patients with AML.

Gilteritinib, developed by Astellas Pharma, has demonstrated inhibitory activity against FLT3 internal tandem duplication (ITD) and FLT3 tyrosine kinase domain.

The FDA’s approval of gilteritinib was based on an interim analysis of the ADMIRAL trial (NCT02421939).

The trial enrolled adults with relapsed or refractory AML who had a FLT3 ITD, D835, or I836 mutation, according to the LeukoStrat CDx FLT3 Mutation Assay.

Patients received gilteritinib at 120 mg daily until they developed unacceptable toxicity or did not show a clinical benefit.

Efficacy results are available for 138 patients, with a median follow-up of 4.6 months (range, 2.8 to 15.8).

The complete response (CR) rate was 11.6% (16/138), the rate of CR with partial hematologic recovery (CRh) was 9.4% (13/138), and the rate of CR/CRh was 21% (29/138).

The median duration of CR/CRh was 4.6 months.

There were 106 patients who were transfusion-dependent at baseline, and 33 of these patients (31.1%) became transfusion-independent during the post-baseline period.

Seventeen of the 32 patients (53.1%) who were transfusion-independent at baseline remained transfusion-independent.

Safety results are available for 292 patients. The median duration of exposure to gilteritinib in this group was 3 months (range, 0.1 to 42.8).

The most common adverse events were myalgia/arthralgia (42%), transaminase increase (41%), fatigue/malaise (40%), fever (35%), noninfectious diarrhea (34%), dyspnea (34%), edema (34%), rash (30%), pneumonia (30%), nausea (27%), constipation (27%), stomatitis (26%), cough (25%), headache (21%), hypotension (21%), dizziness (20%), and vomiting (20%).

Eight percent of patients (n=22) discontinued gilteritinib due to adverse events. The most common were pneumonia (2%), sepsis (2%), and dyspnea (1%).

For more details on the ADMIRAL trial and gilteritinib, see the full prescribing information.

Astellas Pharma

The U.S. Food and Drug Administration (FDA) has approved gilteritinib (Xospata) for use in adults who have relapsed or refractory acute myeloid leukemia (AML) with a FLT3 mutation, as detected by an FDA-approved test.

The FDA also expanded the approved indication for the LeukoStrat CDx FLT3 Mutation Assay to include use with gilteritinib.

The LeukoStrat CDx FLT3 Mutation Assay, developed by Invivoscribe Technologies, Inc., is used to detect FLT3 mutations in patients with AML.

Gilteritinib, developed by Astellas Pharma, has demonstrated inhibitory activity against FLT3 internal tandem duplication (ITD) and FLT3 tyrosine kinase domain.

The FDA’s approval of gilteritinib was based on an interim analysis of the ADMIRAL trial (NCT02421939).

The trial enrolled adults with relapsed or refractory AML who had a FLT3 ITD, D835, or I836 mutation, according to the LeukoStrat CDx FLT3 Mutation Assay.

Patients received gilteritinib at 120 mg daily until they developed unacceptable toxicity or did not show a clinical benefit.

Efficacy results are available for 138 patients, with a median follow-up of 4.6 months (range, 2.8 to 15.8).

The complete response (CR) rate was 11.6% (16/138), the rate of CR with partial hematologic recovery (CRh) was 9.4% (13/138), and the rate of CR/CRh was 21% (29/138).

The median duration of CR/CRh was 4.6 months.

There were 106 patients who were transfusion-dependent at baseline, and 33 of these patients (31.1%) became transfusion-independent during the post-baseline period.

Seventeen of the 32 patients (53.1%) who were transfusion-independent at baseline remained transfusion-independent.

Safety results are available for 292 patients. The median duration of exposure to gilteritinib in this group was 3 months (range, 0.1 to 42.8).

The most common adverse events were myalgia/arthralgia (42%), transaminase increase (41%), fatigue/malaise (40%), fever (35%), noninfectious diarrhea (34%), dyspnea (34%), edema (34%), rash (30%), pneumonia (30%), nausea (27%), constipation (27%), stomatitis (26%), cough (25%), headache (21%), hypotension (21%), dizziness (20%), and vomiting (20%).

Eight percent of patients (n=22) discontinued gilteritinib due to adverse events. The most common were pneumonia (2%), sepsis (2%), and dyspnea (1%).

For more details on the ADMIRAL trial and gilteritinib, see the full prescribing information.

Photo by Bill Branson

The U.S. Food and Drug Administration (FDA) has approved a biosimilar rituximab product for the treatment of non-Hodgkin lymphoma (NHL).

Celltrion’s Truxima (rituximab-abbs) is a biosimilar of Genentech’s Rituxan and the first biosimilar approved in the United States to treat NHL.

Truxima (formerly CT-P10) is approved to treat adults with CD20-positive, B-cell NHL, either as a single agent or in combination with chemotherapy.

Specifically, Truxima is approved as a single agent to treat relapsed or refractory, low grade or follicular, CD20-positive, B-cell NHL.

Truxima is approved in combination with first-line chemotherapy to treat previously untreated follicular, CD20-positive, B-cell NHL.

Truxima is approved as single-agent maintenance therapy in patients with follicular, CD20-positive, B-cell NHL who achieve a complete or partial response to a rituximab product in combination with chemotherapy.

And Truxima is approved as a single agent to treat non-progressing, low-grade, CD20­-positive, B-cell NHL after first-line treatment with cyclophosphamide, vincristine, and prednisone.

The label for Truxima contains a boxed warning detailing the risk of fatal infusion reactions, severe skin and mouth reactions (some with fatal outcomes), hepatitis B virus reactivation that may cause serious liver problems (including liver failure and death), and progressive multifocal leukoencephalopathy.

The FDA said its approval of Truxima is “based on a review of evidence that included extensive structural and functional characterization, animal study data, human pharmacokinetic data, clinical immunogenicity data, and other clinical data that demonstrates Truxima is biosimilar to Rituxan.”

A phase 3 trial recently published in The Lancet Haematology suggested that Truxima is equivalent to the reference product in patients with low-tumor-burden follicular lymphoma.

For more details on Truxima, see the prescribing information.

Photo by Bill Branson

The U.S. Food and Drug Administration (FDA) has approved a biosimilar rituximab product for the treatment of non-Hodgkin lymphoma (NHL).

Celltrion’s Truxima (rituximab-abbs) is a biosimilar of Genentech’s Rituxan and the first biosimilar approved in the United States to treat NHL.

Truxima (formerly CT-P10) is approved to treat adults with CD20-positive, B-cell NHL, either as a single agent or in combination with chemotherapy.

Specifically, Truxima is approved as a single agent to treat relapsed or refractory, low grade or follicular, CD20-positive, B-cell NHL.

Truxima is approved in combination with first-line chemotherapy to treat previously untreated follicular, CD20-positive, B-cell NHL.

Truxima is approved as single-agent maintenance therapy in patients with follicular, CD20-positive, B-cell NHL who achieve a complete or partial response to a rituximab product in combination with chemotherapy.

And Truxima is approved as a single agent to treat non-progressing, low-grade, CD20­-positive, B-cell NHL after first-line treatment with cyclophosphamide, vincristine, and prednisone.

The label for Truxima contains a boxed warning detailing the risk of fatal infusion reactions, severe skin and mouth reactions (some with fatal outcomes), hepatitis B virus reactivation that may cause serious liver problems (including liver failure and death), and progressive multifocal leukoencephalopathy.

The FDA said its approval of Truxima is “based on a review of evidence that included extensive structural and functional characterization, animal study data, human pharmacokinetic data, clinical immunogenicity data, and other clinical data that demonstrates Truxima is biosimilar to Rituxan.”

A phase 3 trial recently published in The Lancet Haematology suggested that Truxima is equivalent to the reference product in patients with low-tumor-burden follicular lymphoma.

For more details on Truxima, see the prescribing information.

Photo by Bill Branson

The U.S. Food and Drug Administration (FDA) has approved a biosimilar rituximab product for the treatment of non-Hodgkin lymphoma (NHL).

Celltrion’s Truxima (rituximab-abbs) is a biosimilar of Genentech’s Rituxan and the first biosimilar approved in the United States to treat NHL.

Truxima (formerly CT-P10) is approved to treat adults with CD20-positive, B-cell NHL, either as a single agent or in combination with chemotherapy.

Specifically, Truxima is approved as a single agent to treat relapsed or refractory, low grade or follicular, CD20-positive, B-cell NHL.

Truxima is approved in combination with first-line chemotherapy to treat previously untreated follicular, CD20-positive, B-cell NHL.

Truxima is approved as single-agent maintenance therapy in patients with follicular, CD20-positive, B-cell NHL who achieve a complete or partial response to a rituximab product in combination with chemotherapy.

And Truxima is approved as a single agent to treat non-progressing, low-grade, CD20­-positive, B-cell NHL after first-line treatment with cyclophosphamide, vincristine, and prednisone.

The label for Truxima contains a boxed warning detailing the risk of fatal infusion reactions, severe skin and mouth reactions (some with fatal outcomes), hepatitis B virus reactivation that may cause serious liver problems (including liver failure and death), and progressive multifocal leukoencephalopathy.

The FDA said its approval of Truxima is “based on a review of evidence that included extensive structural and functional characterization, animal study data, human pharmacokinetic data, clinical immunogenicity data, and other clinical data that demonstrates Truxima is biosimilar to Rituxan.”

A phase 3 trial recently published in The Lancet Haematology suggested that Truxima is equivalent to the reference product in patients with low-tumor-burden follicular lymphoma.

For more details on Truxima, see the prescribing information.

Photo from Bayer

The European Commission (EC) has approved damoctocog alfa pegol (Jivi®), a recombinant human factor VIII therapy.

Damoctocog alfa pegol (formerly BAY94-9027) is approved for the treatment and prophylaxis of bleeding in previously treated patients age 12 and older who have hemophilia A.

The approval is valid in the European Union, Norway, Iceland, and Liechtenstein.

The EC’s decision to approve damoctocog alfa pegol is supported by the phase 2/3 PROTECT VIII trial.

Some results from this trial were published in the Journal of Thrombosis and Haemostasis in 2016. Additional results are available in the U.S. prescribing information for Jivi.

Study design

PROTECT VIII enrolled previously treated adults and adolescents (ages 12 to 65) with severe hemophilia A.

In part A, researchers evaluated different dosing regimens for damoctocog alfa pegol used as prophylaxis and on-demand treatment. An optional extension study was available to patients who completed part A.

In part B, researchers evaluated damoctocog alfa pegol for perioperative management.

In part A, there were 132 patients in the intent‐to‐treat population—112 in the prophylaxis group and 20 in the on-demand group.

Patients received damoctocog alfa pegol for 36 weeks. For the first 10 weeks, patients in the prophylaxis group received twice-weekly dosing at 25 IU/kg.

Patients with more than one bleed during this time went on to receive 30–40 IU/kg twice weekly. Patients with one or fewer bleeds were eligible for randomization to dosing every 5 days (45–60 IU/kg) or every 7 days (60 IU/kg).

Efficacy

The median annualized bleeding rate (ABR) was 4.1 for the patients who were treated twice weekly and were not eligible for randomization (n=13) and 1.9 for patients who were eligible for randomization but continued on twice-weekly treatment (n=11).

The median ABR was 1.9 for patients who were randomized to treatment every 5 days (n=43) and 0.96 for patients who completed prophylaxis with dosing every 7 days (32/43).

The median ABR for patients treated on demand was 24.1.

There were 388 treated bleeds in the on-demand group and 317 treated bleeds in the prophylaxis group. Overall, 73.3% of responses to treatment were considered “excellent” or “good,” 23.3% were “moderate,” and 3.3% were “poor.”

There were 17 patients who underwent 20 major surgeries in part B or the extension study and 10 patients who underwent minor surgeries in part A.

Damoctocog alfa pegol provided “good” or “excellent” hemostatic control during all surgeries.

Safety

Safety data are available for 148 patients age 12 and older.

Adverse events in these patients included abdominal pain (3%), nausea (5%), vomiting (3%), injection site reactions (1%), pyrexia (5%), hypersensitivity (2%), dizziness (2%), headache (14%), insomnia (3%), cough (7%), erythema (1%), pruritus (1%), rash (2%), and flushing (1%).

A factor VIII inhibitor was reported in one adult patient, but repeat testing did not confirm the report.

One adult with asthma had a clinical hypersensitivity reaction and a transient increase of IgM anti-PEG antibody titer, which was negative upon retesting.

Photo from Bayer

The European Commission (EC) has approved damoctocog alfa pegol (Jivi®), a recombinant human factor VIII therapy.

Damoctocog alfa pegol (formerly BAY94-9027) is approved for the treatment and prophylaxis of bleeding in previously treated patients age 12 and older who have hemophilia A.

The approval is valid in the European Union, Norway, Iceland, and Liechtenstein.

The EC’s decision to approve damoctocog alfa pegol is supported by the phase 2/3 PROTECT VIII trial.

Some results from this trial were published in the Journal of Thrombosis and Haemostasis in 2016. Additional results are available in the U.S. prescribing information for Jivi.

Study design

PROTECT VIII enrolled previously treated adults and adolescents (ages 12 to 65) with severe hemophilia A.

In part A, researchers evaluated different dosing regimens for damoctocog alfa pegol used as prophylaxis and on-demand treatment. An optional extension study was available to patients who completed part A.

In part B, researchers evaluated damoctocog alfa pegol for perioperative management.

In part A, there were 132 patients in the intent‐to‐treat population—112 in the prophylaxis group and 20 in the on-demand group.

Patients received damoctocog alfa pegol for 36 weeks. For the first 10 weeks, patients in the prophylaxis group received twice-weekly dosing at 25 IU/kg.

Patients with more than one bleed during this time went on to receive 30–40 IU/kg twice weekly. Patients with one or fewer bleeds were eligible for randomization to dosing every 5 days (45–60 IU/kg) or every 7 days (60 IU/kg).

Efficacy

The median annualized bleeding rate (ABR) was 4.1 for the patients who were treated twice weekly and were not eligible for randomization (n=13) and 1.9 for patients who were eligible for randomization but continued on twice-weekly treatment (n=11).

The median ABR was 1.9 for patients who were randomized to treatment every 5 days (n=43) and 0.96 for patients who completed prophylaxis with dosing every 7 days (32/43).

The median ABR for patients treated on demand was 24.1.

There were 388 treated bleeds in the on-demand group and 317 treated bleeds in the prophylaxis group. Overall, 73.3% of responses to treatment were considered “excellent” or “good,” 23.3% were “moderate,” and 3.3% were “poor.”

There were 17 patients who underwent 20 major surgeries in part B or the extension study and 10 patients who underwent minor surgeries in part A.

Damoctocog alfa pegol provided “good” or “excellent” hemostatic control during all surgeries.

Safety

Safety data are available for 148 patients age 12 and older.

Adverse events in these patients included abdominal pain (3%), nausea (5%), vomiting (3%), injection site reactions (1%), pyrexia (5%), hypersensitivity (2%), dizziness (2%), headache (14%), insomnia (3%), cough (7%), erythema (1%), pruritus (1%), rash (2%), and flushing (1%).

A factor VIII inhibitor was reported in one adult patient, but repeat testing did not confirm the report.

One adult with asthma had a clinical hypersensitivity reaction and a transient increase of IgM anti-PEG antibody titer, which was negative upon retesting.

Photo from Bayer

The European Commission (EC) has approved damoctocog alfa pegol (Jivi®), a recombinant human factor VIII therapy.

Damoctocog alfa pegol (formerly BAY94-9027) is approved for the treatment and prophylaxis of bleeding in previously treated patients age 12 and older who have hemophilia A.

The approval is valid in the European Union, Norway, Iceland, and Liechtenstein.

The EC’s decision to approve damoctocog alfa pegol is supported by the phase 2/3 PROTECT VIII trial.

Some results from this trial were published in the Journal of Thrombosis and Haemostasis in 2016. Additional results are available in the U.S. prescribing information for Jivi.

Study design

PROTECT VIII enrolled previously treated adults and adolescents (ages 12 to 65) with severe hemophilia A.

In part A, researchers evaluated different dosing regimens for damoctocog alfa pegol used as prophylaxis and on-demand treatment. An optional extension study was available to patients who completed part A.

In part B, researchers evaluated damoctocog alfa pegol for perioperative management.

In part A, there were 132 patients in the intent‐to‐treat population—112 in the prophylaxis group and 20 in the on-demand group.

Patients received damoctocog alfa pegol for 36 weeks. For the first 10 weeks, patients in the prophylaxis group received twice-weekly dosing at 25 IU/kg.

Patients with more than one bleed during this time went on to receive 30–40 IU/kg twice weekly. Patients with one or fewer bleeds were eligible for randomization to dosing every 5 days (45–60 IU/kg) or every 7 days (60 IU/kg).

Efficacy

The median annualized bleeding rate (ABR) was 4.1 for the patients who were treated twice weekly and were not eligible for randomization (n=13) and 1.9 for patients who were eligible for randomization but continued on twice-weekly treatment (n=11).

The median ABR was 1.9 for patients who were randomized to treatment every 5 days (n=43) and 0.96 for patients who completed prophylaxis with dosing every 7 days (32/43).

The median ABR for patients treated on demand was 24.1.

There were 388 treated bleeds in the on-demand group and 317 treated bleeds in the prophylaxis group. Overall, 73.3% of responses to treatment were considered “excellent” or “good,” 23.3% were “moderate,” and 3.3% were “poor.”

There were 17 patients who underwent 20 major surgeries in part B or the extension study and 10 patients who underwent minor surgeries in part A.

Damoctocog alfa pegol provided “good” or “excellent” hemostatic control during all surgeries.

Safety

Safety data are available for 148 patients age 12 and older.

Adverse events in these patients included abdominal pain (3%), nausea (5%), vomiting (3%), injection site reactions (1%), pyrexia (5%), hypersensitivity (2%), dizziness (2%), headache (14%), insomnia (3%), cough (7%), erythema (1%), pruritus (1%), rash (2%), and flushing (1%).

A factor VIII inhibitor was reported in one adult patient, but repeat testing did not confirm the report.

One adult with asthma had a clinical hypersensitivity reaction and a transient increase of IgM anti-PEG antibody titer, which was negative upon retesting.

ORLANDO – More stringent gun laws are linked to reduced firearm-related pediatric injury and mortality, and laws restricting children’s access to firearms are linked to reduced pediatric firearm suicide rates, according to research.

Bytmonas/ThinkStock

“State-level legislation could play an important role in reducing pediatric firearm-related deaths,” concluded Jordan S. Taylor, MD, of Stanford (Calif.) University and his colleagues.

Dr. Taylor earned top honors among the American Academy of Pediatrics (AAP) Council on Injury, Violence and Poison Prevention research abstracts when he presented his findings at the annual meeting of the American Academy of Pediatrics.

Firearm injuries account for the second leading cause of death among U.S. children: 3,155 youth ages 19 years and younger died from gunshot injuries in 2016, and more than 17,000 were injured. Yet state laws governing the purchase, ownership, carriage, and storage of guns vary widely across the country. Dr. Taylor and his colleagues conducted two studies to assess the effects of firearm legislation on firearm-related injuries and deaths in U.S. children.

In their first study, they analyzed pediatric inpatient admissions for firearm injuries in 2012 relative to the stringency of state firearm legislation. They relied on five data sources for the analysis: the Kids’ Inpatient Database (KID), the Healthcare Cost and Utilization Project, the Agency for Healthcare Research and Quality, the U.S. Census Bureau, and the 2013 Brady scorecard.

The Brady scorecard provides scores for each state based on the presence and strictness of firearm-related laws, including legislation on background checks, ability of dangerous individuals to purchase guns, trafficking laws, and laws governing the sales, carrying, and purchasing of firearms.

The 10 states with the strictest laws (highest Brady scores) are California, Connecticut, Delaware, Hawaii, Illinois, Maryland, Massachusetts, New Jersey, New York, and Rhode Island. The 10 states with the lowest scores (least-strict legislation) are Alaska, Arizona, Arkansas, Florida, Kentucky, Louisiana, Montana, Nevada, Virginia, and Wyoming.

Among the 6,941 youth (aged 0-20 years) hospitalized in 2012 for firearm injuries, 7% died. More than a third of these (36%) occurred in the South, 25% in the Midwest, 22% in the West, and 17% in the Northeast.

Children most likely to be injured were boys, older children, black and Latino children, and children living in low-income zip codes.

The Midwest and South, which have lower average Brady scores (more lax legislation on guns), had 8.30 injuries per 100,000 children, compared with 7.54 injuries per 100,000 children in the Northeast and West, which have higher average Brady scores (more stringent gun laws). This was a difference of 0.76 injuries per 100,000 children (95% confidence interval, 0.38-1.13; P less than 0.001).

Then the researchers conducted a second analysis that looked specifically at firearm mortality within the context of both child access prevention (CAP) laws and states’ Brady scores. CAP laws include safe storage laws and gun lock laws, for example.

This analysis used the Web-Based Injury Statistics Query and Reporting System to capture pediatric firearm deaths from 2014-2015 and compared these to the 2014 Brady scores and CAP laws.

An estimated 2,715 child gun deaths occurred during the study period, of which 62% were homicides and 31% were suicides. The researchers identified “a significant negative correlation between states’ firearm legislation stringency and pediatric firearm mortality (Spearman correlation coefficient = –0.66) and between presence of CAP laws and firearm suicide rates (Spearman correlation coefficient = –0.56).”

Dr. Taylor said in an interview, “states that have both types of child access prevention laws [had] suicide rates four times lower than states that did not have either of those.”

Positive correlations also showed up between unemployment rate and firearm homicide rate (Spearman correlation coefficient = 0.55) and teen tobacco use and firearm suicide rate (Spearman correlation coefficient = 0.50).

The association between Brady scores and pediatric mortality from firearms remained significant after adjustment for poverty, unemployment, and substance abuse (P less than .01). Similarly, the association between the pediatric firearm suicide rate and CAP laws remained significant after controlling for socioeconomic factors and other firearm legislation (P less than .01).

In a video interview, Dr. Taylor discussed his research findings and their importance in clinical practice.

“It’s absolutely important for pediatricians to talk to families about firearms in their home and also in the homes of their friends that they visit,” Dr. Taylor said. “We try to approach it as a public health issue similar to seat belts and car seats.”

No external funding was used, and Dr. Taylor reported no conflicts of interest.

ORLANDO – More stringent gun laws are linked to reduced firearm-related pediatric injury and mortality, and laws restricting children’s access to firearms are linked to reduced pediatric firearm suicide rates, according to research.

Bytmonas/ThinkStock

“State-level legislation could play an important role in reducing pediatric firearm-related deaths,” concluded Jordan S. Taylor, MD, of Stanford (Calif.) University and his colleagues.

Dr. Taylor earned top honors among the American Academy of Pediatrics (AAP) Council on Injury, Violence and Poison Prevention research abstracts when he presented his findings at the annual meeting of the American Academy of Pediatrics.

Firearm injuries account for the second leading cause of death among U.S. children: 3,155 youth ages 19 years and younger died from gunshot injuries in 2016, and more than 17,000 were injured. Yet state laws governing the purchase, ownership, carriage, and storage of guns vary widely across the country. Dr. Taylor and his colleagues conducted two studies to assess the effects of firearm legislation on firearm-related injuries and deaths in U.S. children.

In their first study, they analyzed pediatric inpatient admissions for firearm injuries in 2012 relative to the stringency of state firearm legislation. They relied on five data sources for the analysis: the Kids’ Inpatient Database (KID), the Healthcare Cost and Utilization Project, the Agency for Healthcare Research and Quality, the U.S. Census Bureau, and the 2013 Brady scorecard.

The Brady scorecard provides scores for each state based on the presence and strictness of firearm-related laws, including legislation on background checks, ability of dangerous individuals to purchase guns, trafficking laws, and laws governing the sales, carrying, and purchasing of firearms.

The 10 states with the strictest laws (highest Brady scores) are California, Connecticut, Delaware, Hawaii, Illinois, Maryland, Massachusetts, New Jersey, New York, and Rhode Island. The 10 states with the lowest scores (least-strict legislation) are Alaska, Arizona, Arkansas, Florida, Kentucky, Louisiana, Montana, Nevada, Virginia, and Wyoming.

Among the 6,941 youth (aged 0-20 years) hospitalized in 2012 for firearm injuries, 7% died. More than a third of these (36%) occurred in the South, 25% in the Midwest, 22% in the West, and 17% in the Northeast.

Children most likely to be injured were boys, older children, black and Latino children, and children living in low-income zip codes.

The Midwest and South, which have lower average Brady scores (more lax legislation on guns), had 8.30 injuries per 100,000 children, compared with 7.54 injuries per 100,000 children in the Northeast and West, which have higher average Brady scores (more stringent gun laws). This was a difference of 0.76 injuries per 100,000 children (95% confidence interval, 0.38-1.13; P less than 0.001).

Then the researchers conducted a second analysis that looked specifically at firearm mortality within the context of both child access prevention (CAP) laws and states’ Brady scores. CAP laws include safe storage laws and gun lock laws, for example.

This analysis used the Web-Based Injury Statistics Query and Reporting System to capture pediatric firearm deaths from 2014-2015 and compared these to the 2014 Brady scores and CAP laws.

An estimated 2,715 child gun deaths occurred during the study period, of which 62% were homicides and 31% were suicides. The researchers identified “a significant negative correlation between states’ firearm legislation stringency and pediatric firearm mortality (Spearman correlation coefficient = –0.66) and between presence of CAP laws and firearm suicide rates (Spearman correlation coefficient = –0.56).”

Dr. Taylor said in an interview, “states that have both types of child access prevention laws [had] suicide rates four times lower than states that did not have either of those.”

Positive correlations also showed up between unemployment rate and firearm homicide rate (Spearman correlation coefficient = 0.55) and teen tobacco use and firearm suicide rate (Spearman correlation coefficient = 0.50).

The association between Brady scores and pediatric mortality from firearms remained significant after adjustment for poverty, unemployment, and substance abuse (P less than .01). Similarly, the association between the pediatric firearm suicide rate and CAP laws remained significant after controlling for socioeconomic factors and other firearm legislation (P less than .01).

In a video interview, Dr. Taylor discussed his research findings and their importance in clinical practice.

“It’s absolutely important for pediatricians to talk to families about firearms in their home and also in the homes of their friends that they visit,” Dr. Taylor said. “We try to approach it as a public health issue similar to seat belts and car seats.”

No external funding was used, and Dr. Taylor reported no conflicts of interest.

ORLANDO – More stringent gun laws are linked to reduced firearm-related pediatric injury and mortality, and laws restricting children’s access to firearms are linked to reduced pediatric firearm suicide rates, according to research.

Bytmonas/ThinkStock

“State-level legislation could play an important role in reducing pediatric firearm-related deaths,” concluded Jordan S. Taylor, MD, of Stanford (Calif.) University and his colleagues.

Dr. Taylor earned top honors among the American Academy of Pediatrics (AAP) Council on Injury, Violence and Poison Prevention research abstracts when he presented his findings at the annual meeting of the American Academy of Pediatrics.

Firearm injuries account for the second leading cause of death among U.S. children: 3,155 youth ages 19 years and younger died from gunshot injuries in 2016, and more than 17,000 were injured. Yet state laws governing the purchase, ownership, carriage, and storage of guns vary widely across the country. Dr. Taylor and his colleagues conducted two studies to assess the effects of firearm legislation on firearm-related injuries and deaths in U.S. children.

In their first study, they analyzed pediatric inpatient admissions for firearm injuries in 2012 relative to the stringency of state firearm legislation. They relied on five data sources for the analysis: the Kids’ Inpatient Database (KID), the Healthcare Cost and Utilization Project, the Agency for Healthcare Research and Quality, the U.S. Census Bureau, and the 2013 Brady scorecard.

The Brady scorecard provides scores for each state based on the presence and strictness of firearm-related laws, including legislation on background checks, ability of dangerous individuals to purchase guns, trafficking laws, and laws governing the sales, carrying, and purchasing of firearms.

The 10 states with the strictest laws (highest Brady scores) are California, Connecticut, Delaware, Hawaii, Illinois, Maryland, Massachusetts, New Jersey, New York, and Rhode Island. The 10 states with the lowest scores (least-strict legislation) are Alaska, Arizona, Arkansas, Florida, Kentucky, Louisiana, Montana, Nevada, Virginia, and Wyoming.

Among the 6,941 youth (aged 0-20 years) hospitalized in 2012 for firearm injuries, 7% died. More than a third of these (36%) occurred in the South, 25% in the Midwest, 22% in the West, and 17% in the Northeast.

Children most likely to be injured were boys, older children, black and Latino children, and children living in low-income zip codes.

The Midwest and South, which have lower average Brady scores (more lax legislation on guns), had 8.30 injuries per 100,000 children, compared with 7.54 injuries per 100,000 children in the Northeast and West, which have higher average Brady scores (more stringent gun laws). This was a difference of 0.76 injuries per 100,000 children (95% confidence interval, 0.38-1.13; P less than 0.001).

Then the researchers conducted a second analysis that looked specifically at firearm mortality within the context of both child access prevention (CAP) laws and states’ Brady scores. CAP laws include safe storage laws and gun lock laws, for example.

This analysis used the Web-Based Injury Statistics Query and Reporting System to capture pediatric firearm deaths from 2014-2015 and compared these to the 2014 Brady scores and CAP laws.

An estimated 2,715 child gun deaths occurred during the study period, of which 62% were homicides and 31% were suicides. The researchers identified “a significant negative correlation between states’ firearm legislation stringency and pediatric firearm mortality (Spearman correlation coefficient = –0.66) and between presence of CAP laws and firearm suicide rates (Spearman correlation coefficient = –0.56).”

Dr. Taylor said in an interview, “states that have both types of child access prevention laws [had] suicide rates four times lower than states that did not have either of those.”

Positive correlations also showed up between unemployment rate and firearm homicide rate (Spearman correlation coefficient = 0.55) and teen tobacco use and firearm suicide rate (Spearman correlation coefficient = 0.50).

The association between Brady scores and pediatric mortality from firearms remained significant after adjustment for poverty, unemployment, and substance abuse (P less than .01). Similarly, the association between the pediatric firearm suicide rate and CAP laws remained significant after controlling for socioeconomic factors and other firearm legislation (P less than .01).

In a video interview, Dr. Taylor discussed his research findings and their importance in clinical practice.

“It’s absolutely important for pediatricians to talk to families about firearms in their home and also in the homes of their friends that they visit,” Dr. Taylor said. “We try to approach it as a public health issue similar to seat belts and car seats.”

No external funding was used, and Dr. Taylor reported no conflicts of interest.